Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy

This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Drug: Erythropoietin

Detailed Description

Cerebral palsy is a disorder of movement and posture resulted from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.

Many experimental animal studies have revealed that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed as follows; neuroprotection effect, angiogenesis, and anti-inflammation.

On the basis of many experimental studies, erythropoietin is suggested as a potential therapy for cerebral palsy.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
      • CHA Bundang Medical Center, CHA University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 3 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Cerebral Palsy
• Abnormal Muscle Tone
• GMFCS (Gross Motor Functional Classification System): II to IV
• Age: 6 months ~ 3 years
• Abnormal Brain MRI compatible to clinical features and non-progressive
• Willing to Comply with All Study Procedure

Exclusion Criteria:

• Known Genetic Disorder
• Baseline Erythropoietin level > 45 mU/mL
• Presence of Drug Hypersensitivity Related to the Study Remedy
• Previous Erythropoietin Treatment before 3 months
• Coagulopathy:

Family History, Unknown Cerebral Infarction, Thromboembolic Events History

• Intractable Seizure Disorder
• Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation
• Uncontrolled Hypertension
• Liver Dysfunction
• Renal Dysfunction
• Absolute Neutrophil Count < 500/dL
• Intracerebral or Intraventricular Hemorrhage
• Malignancy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Erythropoietin and Rehabilitation; recombinant human erythropoietin injection and active rehabilitation	Drug: Erythropoietin; 250 IU/kg, Twice a week for 4 weeks; Other Names: Espogen produced by LG Life Science

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Monitoring of all adverse events during 8 weeks, the study period We selected specially considered adverse events: encephalopathy, intracranial hemorrhage, seizure, hypertension, thromboembolic event, hypoxia, and acute kidney injury. Other adverse events will be recorded. The list was determined by the clinical experience and all adverse reactions reported by the pharmaceutical company.	8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Quality of Movement	GMPM (Gross Motor Performance Measure) as a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones: alignment, coordination, dissociated movement, stability, and weight shift The interrater reliability of GMPM subscores and total scores was 0.758-0.886 (subject n=75, tester n=10).	Baseline - 8 weeks
Changes in Gross Motor Function	GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of 6 sub-scales; lying & rolling, sitting, crawling & kneeling, standing, walking, running & jumping The measured interrater reliability of GMFM subscores and total scores was 0.974 - 0.997 (subject n=101, tester n=10) and intrarater reliability of GMFM subscores and total scores between one most experienced rater and another newly t rained rater was 0.994 - 1.000 (subject n=101, tester n=2).	Baseline - 8 weeks
Changes in Neurodevelopmental Outcomes	K-BSID-II (Korean version of Bayley Scale of Infant Development-II) Motor and Mental scales The measured intrarater and interrater reliability of K-BSID-II motor and mental scales was 0.92 - 0.99 (subject n=55, tester n=10).	Baseline - 8 weeks
Changes in Motor Development	AIMS (Alberta Infant Motor Scale) to assess motor development	Baseline - 8 weeks
Changes in Spasticity	MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord	Baseline - 8 weeks

Collaborators and Investigators

• Sponsor: MinYoung Kim, M.D.
• Collaborators: LG Life Sciences

Study record dates

Study Major Dates

• Study Start: June 1, 2012
• Primary Completion (Actual): October 1, 2013
• Study Completion (Actual): October 1, 2013

Study Registration Dates

• First Submitted: April 23, 2012
• First Submitted That Met QC Criteria: April 25, 2012
• First Posted (Estimate): April 26, 2012

Study Record Updates

• Last Update Posted (Estimate): April 8, 2014
• Last Update Submitted That Met QC Criteria: April 7, 2014
• Last Verified: April 1, 2014

More Information

Terms related to this study

• Keywords: Rehabilitation; Cerebral Palsy; Erythropoietin
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Brain Damage, Chronic; Cerebral Palsy; Paralysis; Hematinics; Epoetin Alfa
• Other Study ID Numbers: CPEPOPhase1